Patients’ and health care providers’ perspectives on quality of hemophilia care in the Netherlands: a questionnaire and interview study

Background Hemophilia care has improved greatly because of advances in treatment options and comprehensive care. In-depth insight into the perspectives of persons with hemophilia and health care providers on their care may provide targets for further improvements. Objectives To assess satisfaction of the hemophilia population with their care, to explore factors determining care satisfaction, and to identify areas for potential health care improvements, including digital health tools. Methods First, to assess care satisfaction and factors determining satisfaction and health care improvements, data from a nationwide, cross-sectional questionnaire among 867 adult and pediatric Dutch persons with hemophilia A or B were analyzed. This included the Hemophilia Patient Satisfaction Scale questionnaire, Canadian Hemophilia Outcomes Kids’ Life Assessment Tool satisfaction questions, a visual analog scale satisfaction score, and open questions. Second, to further explore factors determining satisfaction and health care improvements, semistructured interviews were conducted with 19 persons with hemophilia or their parents and 18 health care providers. Results High care satisfaction was found, with an overall median Hemophilia Patient Satisfaction Scale score of 12 (IQR, 6-21). Participants in the interviews reported that patient-professional interactions, availability of care, and coordination of care were major factors determining satisfaction. Suggested health care improvements included improved information provision and coordination of care, especially shared care with professionals not working within comprehensive care centers. Participants suggested that digital health tools could aid in this. Conclusion Satisfaction with hemophilia care is high among persons with hemophilia in the Netherlands, although several potential improvements have been identified. Accentuating these is especially relevant in the current era of treatment innovations, in which we might focus less on other aspects of care.


| I N T R O D U C T I O N
Hemophilia is an inherited bleeding disorder caused by a deficiency of functional coagulation factor VIII (hemophilia A) or factor IX (hemophilia B). Three types of hemophilia are distinguished: severe hemophilia (defined as a residual clotting factor activity of less than 1%), moderate-severe hemophilia (between 1% and 5%), and mild hemophilia (between 6% and 40%). Severely affected patients have spontaneous joint and muscle bleedings leading to joint damage. As a consequence, life-long prophylactic treatment to prevent bleeds is needed. People regularly self-infuse with coagulation factor products or nonfactor replacement products. Such home treatment with remote advice from health care providers demands high selfmanagement.
Hemophilia care improvements have predominantly been the result of major advances in treatment [1,2]. Additionally, the centralization of Dutch hemophilia care in 6 comprehensive care centers (CCCs) resulted in specialized, multidisciplinary care. Together, this led to improved life expectancy, decreased annual bleeding rate, and improved social participation [3,4].
Yet, people's perception of the quality of their care is not determined by these health outcomes alone. Reported additional elements of care satisfaction are ease of treatment administration, burden of disease, relationships with health care providers, health care costs, information provision, and coordination of care [5][6][7][8][9][10]. Coordination of care is defined as the organization of care activities between 2 or more participants involved in a patient's care, including the patient himself/herself [11]. Additionally, the organization of a health care system is an important determinant of satisfaction [9]. The Dutch health care system is often ranked as one of the highest in the world, as illustrated in Box A [12,13]. Finally, recent advances in digital technology have changed the landscape of health care. As in other medical fields, the COVID-19 pandemic accelerated the use of digital health tools in hemophilia care.
In-depth insight into how people with hemophilia perceive the quality of Dutch hemophilia care may provide targets for further improvement. Therefore, in this study, we aimed to assess perspectives of the Dutch hemophilia population on the quality of their care.
First, we assessed the degree of their satisfaction with hemophilia care. Second, we explored factors determining care satisfaction. Third, we identified areas for potential health care improvements, with a special focus on digital health tools. Patient perspectives were juxtaposed with health care provider perspectives to help determine which improvements are most desired.

| M E T H O D S
This mixed methods study consisted of 2 parts. First, in a quantitative study of nationwide, cross-sectional questionnaire data, we assessed patient satisfaction with hemophilia care, factors determining care satisfaction, and areas for potential health care improvements. Second, in a qualitative study of semistructured interviews, we further explored the latter 2 topics: factors determining care satisfaction and potential health care improvements.
B O X A . Organization of Dutch health care.
Due to a compulsory health insurance, which is provided by insurers acting in competition, the Dutch health care system is relatively efficient and accessible, as compared to others. [13] Coagulation factor costs are covered by public insurance, except from a yearly deductible fee of approximately €400, which is paid by individuals themselves. Children aged of <18 years are insured through their parents. For them, no additional fee is required.

Essentials
• The perceived quality of care by people with hemophilia is determined by different elements.
• We assessed care satisfaction by conducting a nationwide questionnaire and in-depth interviews.
• Satisfaction was mainly determined by care availability, communication, and coordination of care.
• Desired improvements related to information provision, coordination responsibilities, and e-Health.

| Quantitative study
This study was part of the sixth nationwide "Hemophilia in the Netherlands" (HiN6) study, the details of which are published elsewhere [3,4].

| Participants
From June 2018 until July 2019, all male pediatric and adult patients with severe, moderate-severe, and mild hemophilia A or B known in the Netherlands were invited to participate. Parents of children (0-11 years), adolescents (between 12 and 17 years), and adults (18 years and older) completed age-specific questionnaires.

| Measures
The HiN6 questionnaire covered several aspects of hemophilia. For the current study, a subset of data on hemophilia care satisfaction was used. Respondents who completed relevant questions on care satisfaction were included, as illustrated by the study flowchart ( Figure 1).

| Measures -Patient satisfaction with hemophilia care (Hemo-Sat, CHO-KLAT, and VAS scales)
To assess satisfaction among adults and parents of children aged 0 to 11 years, the validated Hemophilia Patient Satisfaction Scale (Hemo-Sat) questionnaire was used [14,15]. Hemo-Sat is a hemophilia-specific care satisfaction questionnaire for adults (version Hemo-SatA, 34 items) and parents of young children (version Hemo-SatP, 35 items).
Hemo-Sat has been validated in 28 languages [16]. Questions are divided into 6 domains: ease and convenience, efficacy, burden, specialist/nurse, center/hospital, and general satisfaction. Answers are given on a 5-point Likert scale, ranging from total agreement to total disagreement on statements. Both total and domain scores were analyzed, as well as individual item responses. Each standardized (domain) score ranged from 0 to 100, with lower scores indicating higher satisfaction. For each Hemo-Sat domain, a minimum data completion rule of 75% was applied. Participants who completed at least 1 Hemo-Sat domain were included. Hemo-Sat's reliability is high (Cronbach's α = 0.71-0.95, assessed among adults) [15,17].
Because Hemo-Sat was not validated for adolescents aged 12 to 17 years, 5 items from the Canadian Hemophilia Outcomes Kids' Life Assessment Tool (CHO-KLAT) were used for the assessment of care satisfaction [18]. CHO-KLAT is a hemophilia-specific questionnaire for children aged 4 to 18 years to evaluate health-related quality of life in the previous 4 weeks. Of 35 items, 5 evaluate care satisfaction. All adolescents who completed at least 1 of these 5 items were included.
Adults and parents of children aged 0 to 11 years were asked the following question: "How would you rate your care in its totality for the past 12 months?" Grades were given on a visual analog scale (VAS) from 1 (lowest) to 10 (highest). The self-reported number of health care providers that participants had been in contact with during the 12 months prior to filling out the questionnaire. f For adults, the highest completed education level is reported. For adolescents, the highest current education level is reported. For adults, the lower education level includes primary education, prevocational secondary education (VMBO), lower secondary vocational training (MBO-1), and the first 3 years of senior general secondary education (HAVO) and preuniversity secondary education (VWO). The intermediate education level includes upper secondary education (HAVO/VWO) and vocational and middle management training (MBO-2, MBO-3, and MBO-4). Higher education includes Bachelor and Master degree programs at universities of applied sciences (HBO), research universities (WO), and doctoral degree programs. For adolescents, the lower education level includes prevocational secondary education (VMBO) and secondary vocational training (MBO). The intermediate education level includes senior general secondary education (HAVO) and vocational and middle management training (MBO). The higher education level includes preuniversity secondary education (VWO). Next, their perspectives on health care improvements were discussed, including perspectives on digital health tools and teleconsulting.

| Data analysis
All interviews were analyzed using MAXQDA. Themes were assessed using a directed form of thematic content analysis because elements of our coding scheme were predetermined by our research question.

| Ethics
Approval was given by the Institutional Review Board of the Amsterdam University Medical Center (W20_383 # 20.428). All participants signed an informed consent form.
The overall questionnaire response rate was 46% (1009 of 2191), as illustrated in the study flowchart ( Figure 1). Respondents were similar to the overall Dutch hemophilia population in terms of age and disease severity [4]. Respondents' education level as well as their socioeconomic participation were similar to those of the overall Dutch population [22,23]. Of respondents, 80% were adults, 6% were adolescents, and 14% were parents of children aged 0 to 11 years.
Relevant data for the current study were available for 867 participants. Participant characteristics are presented in Table 1. Included participants were similar to overall HiN6 respondents in terms of the proportion with severe hemophilia (39% vs 38%), median age (44 vs 40 years), and prophylaxis use (38% vs 36%) [4]. Of study participants with severe hemophilia, 91% used prophylaxis. Of children under the age of 18 years with severe hemophilia, 97% used prophylaxis. Since nonfactor replacement therapy was not readily available at the time of this study, most patients used factor VIII or factor IX prophylaxis.

| Patient satisfaction with hemophilia care
The median (IQR) overall Hemo-Sat score of 704 adults and parents of children aged 0 to 11 years was 12 (6-21). The results are shown in Figure 2. Values are presented in Supplementary Table 1. The highest satisfaction was expressed on the 3 Hemo-Sat domains: "general satisfaction," "specialist/nurse," and "center/hospital." Overall, 96% (737 of 764) of adults and parents reported that they were satisfied or very satisfied with their care. Lower satisfaction was expressed on the domains "efficacy," "burden," and "ease and convenience." Of adults and parents, 63% (446 of 711) reported that they were (very) confident that adequate prophylaxis administration could prevent bleeds and 12% (99 of 797) reported that treatment (strongly) interfered with everyday life. On the domain "burden,"  Adults rated their care with a mean VAS score of 8 (95% CI, 6-10; n = 573). For 99 parents of young children aged 0 to 11 years, this was 8 (95% CI, 7-10).

| Factors determining care satisfaction
In total, 449 participants responded to open questions. Many patients described their long-lasting bonds with health care providers ( Table 2, quotes Q1 and Q2). Seventy-nine adults argued that health care costs were too high. The majority (90%, 71 of 79) referred to the yearly recurrent individual deductible fee, which they considered unjustly high for chronically ill patients (Q3). A minority (10%) referred to the In describing care improvements, 53 adults and parents expressed a desire to be more frequently and extensively informed on novel treatment options (Q8). Several patients suggested that digital health tools could assist with this. Some patients suggested that improvements in health information exchange would aid the sometimes lacking communication between professionals working within and outside CCCs. Finally, teleconsulting was suggested to further increase the approachability of care institutions.
Characteristics are presented in Table 3.
T A B L E 2 Questionnaire quotes on elements determining hemophilia care satisfaction.  [25]. In a German questionnaire study, 96% of participants were either satisfied or very satisfied with their care [26].
In the questionnaire study, 2 health care domains were evaluated most positively by patients: "specialist/nurse" and "center/hospital." These positive aspects related to patient-professional communication, treatment explanations, and availability of care. The domains "efficacy," "burden," and "ease and convenience" were ranked less favorably, primarily due to the high burden associated with repeated intravenous administration of therapy. This finding is supported by previous research. In a Dutch qualitative study, interviewed patients said that self-administering treatment can be challenging [27]. In a mixed methods study among German persons with hemophilia and their parents, 34% would switch to a new product for easier Q19 "Of course, the problem is that no one is ultimately responsible for a patient in contact with multiple doctors."-Male patient, aged >65 y, with severe hemophilia A.
Q20 "Everybody knows that currently, the exchange of medical information is a mess… is in need of improvement.
[…] This is the biggest bottleneck when talking about the quality of care at this moment."-Hematologist with 0-10 y of experience.
BRANDS ET AL.
-9 of 14 administration [28]. However, since nonfactor replacement therapy was not readily available at the time of this study, this identified burden is expected to improve [4].
To date, no studies have compared satisfaction among adults and parents. We found that children worry more about receiving injections compared to adults. We suggest 2 hypotheses. First, it is known that fear of needles is more prevalent among young children [29]. Second, older participants might consider current treatment administration easier in comparison to the past and thus express higher satisfaction [27].
Finally, the observed differences in Hemo-Sat domain scores between subgroups with different treatment regimens and hemophilia severity suggest that different patients experience the organization of their care differently and have different needs. Albeit a logical and perhaps inevitable consequence, this creates a remarkable paradox. To compensate for the lack of knowledge among non-CCC professionals, patients are expected to be alert and take on a coordinating role, especially those in contact with many different health care providers. Patients often expressed resistance toward this role but also indicated a certain habituation in doing so.

| Health care improvements
Two potential health care improvements were suggested by participants. Stressing these additional care improvements is especially B O X C . Themes regarding current and potential future digital health tools.

| Strengths and limitations
A strength of this study is the combination of quantitative and qualitative methods. This enabled us to focus interview questions on the topics identified from the questionnaire. The first limitation of the questionnaire study is the potential selection of patients who completed at least 1 Hemo-Sat domain. This may have led to an overestimation of treatment satisfaction because these patients might be more involved in their care than the general population. We were unable to report race or ethnicity as a sociocultural determinant of health but did report education level. Second, Hemo-Sat has not been validated for children aged between 12 and 17 years, resulting in a partial evaluation of pediatric care. Third, in general, patients tend to report high satisfaction with their health care providers, which partly impedes critical judgments [33] Inclusiveness of care Q23 "For regular outpatient clinic appointments, I believe video conferencing is inferior to seeing patients in person. You receive less information. Especially for those patients where, I believe, there is a discrepancy in the perception of whether things are going well. And by that, naturally, I refer to our adolescent patients."-Hematologist with >20 y of experience.

Q24
"The danger in these kinds of applications… the biggest danger is that we are only improving care for those that already have good access to it. The more e-Health tools we develop, and the fancier they become, the more this may become apparent."-Psychologist with 0-10 y of experience.

Q27
"You'd actually want to have a scoring system that characterizes a relationship. The value, which defines the quality of the patient-professional relationship… that value should be higher than a certain threshold level, before you can switch to video consultations.
[…] And one patient matched with one professional reaches this threshold faster than another set."-Pediatric hematologist with >20 y of experience.
Technical and usability preconditions Q28 "Image I need six screens when talking to a patient? I wouldn't even be able to see my patients; they would sit behind those screens! When I would need to check all of those [apps and tools], I wouldn't even be looking at my patient anymore. We should be cautious… we should aim to integrate things."-Hematologist with >20 y of experience.
BRANDS ET AL.

ACKNOWLEDGMENTS
We thank all participants of the questionnaire study and interviews.
We also thank the Dutch Ministry of Health and the Dutch Hemophilia Foundation (Stichting Haemophilia) for their support.

ETHICS STATEMENT
For the quantitative study, approval was obtained from the Institutional Review Board of the Leiden University Medical Center (NL59114.058.17).
Participants who completed the questionnaire were considered to consent. For the qualitative study, approval was given by the Institutional

RELATIONSHIP DISCLOSURE
The HemoNED Foundation received a grant/research support from SPRL. For more information, visit www.symphonyconsortium.nl. S.C.G.
reports an unrestricted research grant from Sobi. The remaining authors have no competing interest to disclose.

DATA AVAILABILITY
The datasets generated during and/or analyzed during the current study are available from the corresponding author on reasonable request.